Hetanshi Naik, PhD^1*, Manisha Balwani, MD^2*, Hilary H Colwell, MPH^3*, Susan D Mathias, MPH^3*, Kristen Wheeden, MBA^4*, Chelsea Norregaard, PhD, MPH^5*, Melanie Chin, PhD^5* and William Savage, MD, PhD⁵

¹Stanford University, Palo Alto, CA
²Icahn School of Medicine At Mount Sinai, New York, NY
³Health Outcomes Solutions, Palm Beach Gardens, FL
⁴United Porphyrias Association, Bethesda, MD
⁵Disc Medicine, Watertown, MA

Kevin Chien^1*, Lindsay Cowley, MA^2*, Daniel Warner, BScH, CCRP^2*, Derek Little, MD^1*, Margaret Ostrowski^3*, Carol West^3*, Roy Khalife, MD, MHPE, FRCPC^2,4 and Deborah Siegal, MD, MSc^2,5

¹Department of Medicine, McMaster University, Hamilton, ON, Canada
²Ottawa Hospital Research Institute, Ottawa, ON, Canada
³CanVECTOR, Montreal, QC, Canada
⁴Department of Medicine, University of Ottawa, The Ottawa Hospital, Ottawa, ON, Canada
⁵Department of Medicine, University of Ottawa, Ottawa, ON, Canada

Anna L. Parks, MD¹, Rushad Patel, MBBS², Dana Elizabeth Angelini, MD³, Jordan K Schaefer, MD, MSc⁴, Ang Li, MD, MS⁵, Alejandra Gutierrez Bernal, MD^6*, Shruti Chaturvedi, MBBS⁷, Anjlee Mahajan, MD⁸, Leslie L Lake, BA^9* and Jori E. May, MD¹⁰

¹Department of Medicine, Division of Hematology & Hematologic Malignancies, University of Utah, Salt Lake City, UT
²BIDMC, Boston, MA
³Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
⁴Division of Hematology/Oncology, Department of Internal Medicine, University of Michigan, Ann Arbor, MI
⁵Baylor College of Medicine, Houston, TX
⁶University of Minnesota, Minneapolis
⁷Division of Hematology, Department of Medicine, Johns Hopkins Hospital, BALTIMORE, MD
⁸University of California, Davis, Sacramento, CA
⁹National Blood Clot Alliance, New York, NY
¹⁰Department of Medicine, Division of Hematology/Oncology, University of Alabama at Birmingham, Birmingham, AL

Roy Khalife, MD, MHPE, FRCPC^1,2, Lindsay Cowley, MA^1*, Alan T. Tinmouth, MD, MSc^1,3*, Lisa D. Duffett, MD, MSc^1,3*, Vanessa Bourck, RN, BScN, MN^4*, Alfonso Iorio, MD, PhD⁵, Davide Matino, MD, MSc^6*, Kay Decker, RN^7*, Karen Strike, PhD, MScPT, BKinHons^8*, David Page^9*, Natasha Pardy, MD, FRCPC^10*, Jerome M. Teitel, MD¹¹ and Haowei Linda Sun, MD MHSc FRCPC¹²

¹Ottawa Hospital Research Institute, Ottawa, ON, Canada
²Department of Medicine, University of Ottawa, The Ottawa Hospital, Ottawa, ON, Canada
³Department of Medicine, University of Ottawa, Ottawa, ON, Canada
⁴The Ottawa Hospital, Ottawa, ON, Canada
⁵Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada
⁶Thrombosis and Atherosclerosis Research Institute (TaARI), McMaster University, Hamilton, ON, Canada
⁷McMaster Children's Hospital, Hamilton Health Sciences, Hamilton, ON, Canada
⁸Faculty of Health Sciences, McMaster University, Hamilton, ON, Canada
⁹Canadian Hemophilia Society, Montreal, QC, Canada
¹⁰Division of Hematology, Eastern Health, St. John's, NF, Canada
¹¹Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada
¹²Division of Hematology, Department of Medicine, University of Alberta, Edmonton, AB, Canada

Abdelrahman Noureldin, BHSc^1*, Victoria Ivankovic, MD^2*, Mary Farnand, RN/IA, BScN, MN^2*, Celine Dainhi^3*, Rebecca Auer, MD, MSc^2*, Sameer Apte, MD^2*, Lev Bubis, MD^4*, Pamela Hebbard, MD^3*, Justin Rivard, MD^3*, Megan Delisle, MD^3* and Marc Carrier, MD, MSc⁵

¹Department of Medicine, University of Ottawa, Ottawa, Canada
²Department of Surgery, University of Ottawa, Ottawa, Canada
³Cancer Care Manitoba (CCMB), Winnipeg, Canada
⁴Department of Surgery, University of Manitoba, Winnipeg, Canada
⁵Department of Medicine, University of Ottawa at The Ottawa Hospital and the Ottawa Hospital Research Institute, Ottawa, Canada

Azra Borogovac, MD¹, Ellie Moses-Okwei, PhD^2*, Lindsay Gurska, PhD^2*, Emily Zyborowicz, MPH^2*, Shelby Sullivan, PharmD^2*, Jeffrey Carter, PhD^2* and Cherilyn Heggen, PhD^2*

¹Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA
²PRIME Education, New York, NY

Prakhyath Srikaram, Paul S Levy, MD, MBA^* and Charlotte Brewer^*

Baptist Memorial Hospital - North Mississippi, Oxford, MS

Swetha Kambhampati Thiruvengadam, MD¹, Nikhil Thiruvengadam^2*, Steven T. Rosen, MD³, Chadi Nabhan, MD, MBA⁴, Kevin Knopf, MD MBA^5*, Linda Martin, PhD^6*, Kenneth R. Carson, MD, PhD⁷, Peter Georgantopoulos^8*, Gretchen Watson^6*, Edward Smith^9*, Edward Zyszkowski^10*, Sony Jacob^11*, Dennis Banegas, BA^12*, William Hrushesky, MD^6*, Iain Cumming Macdougall, BSc, MBBChir, MD, MRCP, FRCP^13*, Manuel Gonzalez-Brio^14*, David Aboulafia, MD^15*, Oliver Sartor^16*, Robert Peter Gale^17* and Charles Bennett, MD¹¹

¹Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA
²Gastroenterology, Loma Linda University, Loma Linda, CA
³Department of Hematology and HCT, City of Hope Comprehensive Cancer Center, Duarte, CA
⁴University of South Carolina, Deerfield, IL
⁵U of SOuth Carolina, COLUMBIA, SC
⁶U of South Carolina, Columbia
⁷Robert H Lurie Comprehensive Cancer Center, Northwestern University Feinberg School of Medicine, Chicago
⁸U of South Carolina, Columbia, SC
⁹University of South Carolina, Columbia, CA
¹⁰University of South Carolina, Columbia
¹¹University of South Carolina, Columbia, SC
¹²Smart State Center for Medication Safety and Efficacy, University of South Carolina, COLUMBIA, SC
¹³King's College Hospital, London, GBR
¹⁴University of Miami, Miami, FL
¹⁵Virginia Mason Clinic, Seattle
¹⁶Mayo Clinic, Rochester, MN
¹⁷Centre For Haematology Research, Department of Immunology and Inflammation, Imperial College London, London, GBR

Jennifer Heimall^1,2*, Luis Ignacio González Granado^3,4*, Claire Booth, MBBS, PhD^5*, Patrice Carter^6*, Emily Matthews^6*, Maria Chitty-Lopez, MD^7*, Miranda Bailey^7*, Susan E Prockop, MD^8*, Andrew R Gennery, MD^9* and Jennifer W Leiding, MD^10,11,12*

¹Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³Department of Pediatrics, Immunodeficiency Unit, University Hospital 12 de Octubre, Madrid, Spain
⁴School of Medicine, Complutense University, Madrid, Spain
⁵Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital NHS Trust, London, United Kingdom
⁶Health Economics and Outcomes Research Ltd., Cardiff, United Kingdom
⁷Rocket Pharmaceuticals, Inc., Cranbury, NJ
⁸Stem Cell Transplant Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
⁹Translational and Clinical Research Institute, Newcastle University, Newcastle Upon Tyne, United Kingdom
¹⁰Division of Allergy and Immunology, Department of Pediatrics, Johns Hopkins University, Baltimore, MD
¹¹Cancer and Blood Disorders Institute, Johns Hopkins All Children’s Hospital, St. Petersburg, FL
¹²Institute for Clinical and Translational Research, John Hopkins All Children's Hospital, St. Petersburg, FL

Josette M Kamel, MD¹ and Mark Chaitowitz, MD²

¹Internal Medicine, Montefiore Medical Center, The Bronx, NY
²Hematology, Montefiore Medical Center, New York, NY

Ang Li, MD, MS¹, Shengling Ma, MD, PhD², Raka Bandyo, MS^3*, Mrinal Ranjan, MPH^1*, Elizabeth Chiang^4*, Joyce Tiong^1*, Jun Yang Jiang, MD¹, Justine Ryu, MD⁵, Omid Jafari, PhD^1*, Christopher I Amos^1*, Nathanael R. Fillmore, PhD^6*, Jennifer La^6*, Karlyn A. Martin, MD, MS⁷, Chirayu J Shah, MD^1* and Aparna Jotwani, MD^1*

¹Baylor College of Medicine, Houston, TX
²Section of Hematology-Oncology, Baylor College of Medicine, HOUSTON, TX
³Harris Health System, Houston, TX
⁴Baylor College of Medicine, houston, TX
⁵Section of Hematology, Yale University School of Medicine, New Haven, CT
⁶VA Boston Healthcare System, Boston, MA
⁷Division of Hematology and Oncology, University of Vermont Larner College of Medicine, Burlington, VT

Julia Levy, MD^1*, Rabeka Ali, MS^2*, Mandy VanSandt, DO^2*, Shelby Koppinger, Pharm D^3*, Francisco Hernandez-Ilizaliturri, MD^4* and Merav Sendowski, MD⁵

¹Department of Internal Medicine, Oregon Health and Science University, Oregon Health and Science University, Portland, OR
²Department of Pathology and Laboratory Medicine, Oregon Health and Science University, Portland, OR
³Department of Pharmacy, Oregon Health & Science University, Oregon Health and Science University, Portland, OR
⁴Department of Immunology, Roswell Park Comprehensive Cancer Center, Buffalo, NY
⁵Department of Hematology and Oncology, Oregon Health and Science University, Oregon Health and Science University, Portland, OR

Charles Greenberg, MD¹, Kimberley Youkhana, MD, MSCR² and Benjamin Djulbegovic, MD PhD¹

¹Medical University of South Carolina, Charleston, SC
²Division of Hematology and Oncology, Medical University of South Carolina, Charleston, SC

Kunal C Potnis, MD^1*, Satoko Ito, MD, PhD², Kishan Patel, MD^3*, Rory M. Shallis, MD^4,5, Nikolai A. Podoltsev, MD, PhD^2,6, Tariq Kewan, MD⁷, Jessica M. Stempel, MD⁷, Lourdes Mendez, MD^8*, Scott F Huntington^9,10, Maximilian Stahl, MD¹¹, Amer M. Zeidan, MBBS, MHS^5,8, Jan Philipp Bewersdorf, MD^5,8 and George Goshua, MD, MSc^12,13

¹Department of Internal Medicine, Yale School of Medicine, New Haven, CT
²Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT
³Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, USA, New York, NY
⁴Department of Internal Medicine, Section of Hematology, Yale School of Medicine - Yale Cancer Center, Killingworth, CT
⁵Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, CT
⁶Yale Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, New Haven, CT
⁷Department of Internal Medicine, Section of Hematology, Yale University, New Haven, CT
⁸Department of Internal Medicine, Section of Hematology, Yale School of Medicine - Yale Cancer Center, New Haven, CT
⁹Yale School of Medicine, New Haven, CT
¹⁰Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale School of Medicine, New Haven, CT
¹¹Dana Farber Cancer Institute, Boston, MA
¹²Hematology-Oncology & Yale CORE, Yale School of Medicine & Yale Cancer Center, New Haven, CT
¹³Center for Outcomes Research and Evaluation, Yale University and Yale New Haven Hospital, New Haven, CT